Rona Therapeutics Co. Ltd. has announced the submission of RN-5681 to the Australian Human Research Ethics Committee (HREC), and anticipates dosing to begin in a phase I trial in the first quarter of next year.
Corsera Health Inc. has filed a clinical trial notification (CTN) seeking to initiate a phase I trial of COR-1004 in New Zealand. COR-1004 is a novel investigational subcutaneously administered siRNA targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) to reduce LDL cholesterol (LDL-C).
Researchers from the Universitat de Barcelona and Oregon Health & Science University have developed a novel gene silencing technique using polypurine reverse Hoogsteen hairpins (PPRH) to target and inhibit the expression of PCSK9.
With plenty of GLP-1 money to spend, Eli Lilly and Co. is buying Verve Therapeutics Inc. and its gene-editing program for about $1.3 billion. Two of Verve’s one-time treatments are in the clinic. Lead candidate VERVE-102, a gene-editing treatment targeting PCSK9, is in a phase Ib study to reduce cholesterol levels.
Positive early stage data for Verve Therapeutics Inc.’s base editing therapy points to a range of development options, including bringing partner Eli Lilly and Co. in a little closer. The new data helped ease the company’s pain from the April 2 enrollment pause of a similarly designed therapy from Verve. Verve’s Heart-2 phase Ib of VERVE-102 in treating 14 patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease showed one infusion led to dose-dependent decreases in blood PCSK9 protein levels and low density lipoprotein cholesterol.
Epigenetic editing is a promising method for gene regulation in vitro and in vivo, allowing precise control of gene expression without altering the DNA sequence, thereby minimizing genotoxic risks.
A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy of a defective gene was inserted at a precise locus in the genome.
Proprotein convertase subtilisin/kexin type-9 (PCSK9) is highly expressed in adult hepatocytes. PCSK9 binds to and promotes the degradation of the low-density lipoprotein (LDL) receptor, thereby increasing LDL cholesterol levels. PCSK9 inhibition has emerged as a promising strategy for cardiovascular diseases. However, it is still unclear whether PCSK9 can trigger blood vessel inflammation directly modulating monocytes or endothelial cells independently of LDL receptor.
Trouble continues to dog Verve Therapeutics Inc.’s base editor of the PCSK9 gene, VERVE-101, so the company paused enrollment in a phase Ib study in cholesterol lowering to focus on the similarly designed VERVE-102.
Plasma pharmacodynamic biomarkers may be a reliable tool for biosimilarity assessment without having to rely on clinical trials, which are costly and time consuming.
